标题
Cystic Fibrosis Gene Therapy: Looking Back, Looking Forward
作者
关键词
-
出版物
Genes
Volume 9, Issue 11, Pages 538
出版商
MDPI AG
发表日期
2018-11-07
DOI
10.3390/genes9110538
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- A G542X cystic fibrosis mouse model for examining nonsense mutation directed therapies
- (2018) Daniel R. McHugh et al. PLoS One
- Axicabtagene ciloleucel (KTE-C19), an anti-CD19 CAR T therapy for the treatment of relapsed/refractory aggressive B-cell non-Hodgkin’s lymphoma
- (2018) Michael D Jain et al. Therapeutics and Clinical Risk Management
- Widespread airway distribution and short-term phenotypic correction of cystic fibrosis pigs following aerosol delivery of piggyBac/adenovirus
- (2018) Ashley L Cooney et al. NUCLEIC ACIDS RESEARCH
- Tezacaftor for the treatment of cystic fibrosis
- (2018) Marc A. Sala et al. Expert Review of Respiratory Medicine
- Human Bocavirus Type-1 Capsid Facilitates the Transduction of Ferret Airways by Adeno-Associated Virus Genomes
- (2017) Ziying Yan et al. HUMAN GENE THERAPY
- PEGylated composite nanoparticles of PLGA and polyethylenimine for safe and efficient delivery of pDNA to lungs
- (2017) Atul Kolte et al. INTERNATIONAL JOURNAL OF PHARMACEUTICS
- Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
- (2017) Stephen Russell et al. LANCET
- The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients
- (2017) Manzar Ashtari et al. OPHTHALMOLOGY
- Long-Term Expression of the HumanCFTRGene in Mouse Airway via Helper-Dependent Adenoviral Vector Delivery and Transient Immunosuppression
- (2016) Huibi Cao et al. HUMAN GENE THERAPY
- Early pulmonary disease manifestations in cystic fibrosis mice
- (2016) Rebecca J. Darrah et al. Journal of Cystic Fibrosis
- Relationships among CFTR expression, HCO3−secretion, and host defense may inform gene- and cell-based cystic fibrosis therapies
- (2016) Viral S. Shah et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
- (2016) Eric W F W Alton et al. THORAX
- Analysis of gene repair tracts from Cas9/gRNA double-stranded breaks in the human CFTR gene
- (2016) Jennifer A. Hollywood et al. Scientific Reports
- Classification of CFTR mutation classes
- (2016) Fernando Augusto Lima Marson et al. Lancet Respiratory Medicine
- Loss of cftr function leads to pancreatic destruction in larval zebrafish
- (2015) Adam Navis et al. DEVELOPMENTAL BIOLOGY
- Optimization of Recombinant Adeno-Associated Virus-Mediated Expression for Large Transgenes, Using a Synthetic Promoter and Tandem Array Enhancers
- (2015) Ziying Yan et al. HUMAN GENE THERAPY
- Hybrid Nonviral/Viral Vector Systems for Improved piggyBac DNA Transposon In Vivo Delivery
- (2015) Ashley L Cooney et al. MOLECULAR THERAPY
- Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
- (2015) Eric W F W Alton et al. Lancet Respiratory Medicine
- Testing gene therapy vectors in human primary nasal epithelial cultures
- (2015) Huibi Cao et al. Molecular Therapy-Methods & Clinical Development
- TALEN-Mediated Generation and Genetic Correction of Disease-Specific Human Induced Pluripotent Stem Cells
- (2014) Sivaprakash Ramalingam et al. CURRENT GENE THERAPY
- Long-term therapeutic and reporter gene expression in lentiviral vector treated cystic fibrosis mice
- (2014) Patricia Cmielewski et al. JOURNAL OF GENE MEDICINE
- Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia
- (2014) Shannon L. Maude et al. NEW ENGLAND JOURNAL OF MEDICINE
- Characterization of Defects in Ion Transport and Tissue Development in Cystic Fibrosis Transmembrane Conductance Regulator (CFTR)-Knockout Rats
- (2014) Katherine L. Tuggle et al. PLoS One
- Lung Phenotype of Juvenile and Adult Cystic Fibrosis Transmembrane Conductance Regulator–Knockout Ferrets
- (2013) Xingshen Sun et al. AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY
- Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs
- (2013) David A. Stoltz et al. JOURNAL OF CLINICAL INVESTIGATION
- A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis: Table 1
- (2013) Eric W F W Alton et al. THORAX
- Airway gene transfer in a non-human primate: Lentiviral gene expression in marmoset lungs
- (2013) N. Farrow et al. Scientific Reports
- The Rabbit as a Model for Studying Lung Disease and Stem Cell Therapy
- (2013) Nurfatin Asyikhin Kamaruzaman et al. Biomed Research International
- Efficient Gene Delivery to Pig Airway Epithelia and Submucosal Glands Using Helper-Dependent Adenoviral Vectors
- (2013) Huibi Cao et al. Molecular Therapy-Nucleic Acids
- Assessment of F/HN-Pseudotyped Lentivirus as a Clinically Relevant Vector for Lung Gene Therapy
- (2012) Uta Griesenbach et al. AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
- Reduced airway surface pH impairs bacterial killing in the porcine cystic fibrosis lung
- (2012) Alejandro A. Pezzulo et al. NATURE
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- Lentiviral Vector Gene Transfer to Porcine Airways
- (2012) Patrick L Sinn et al. Molecular Therapy-Nucleic Acids
- A Hyperactive Transposase Promotes Persistent Gene Transfer of a piggyBac DNA Transposon
- (2012) Erin R Burnight et al. Molecular Therapy-Nucleic Acids
- Sequential targeting of CFTR by BAC vectors generates a novel pig model of cystic fibrosis
- (2011) N. Klymiuk et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- Cystic fibrosis transmembrane conductance regulator with a shortened R domain rescues the intestinal phenotype of CFTR-/- mice
- (2011) L. S. Ostedgaard et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809
- (2011) F. Van Goor et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Readministration of helper-dependent adenoviral vectors to mouse airway mediated via transient immunosuppression
- (2010) H Cao et al. GENE THERAPY
- Disease phenotype of a ferret CFTR-knockout model of cystic fibrosis
- (2010) Xingshen Sun et al. JOURNAL OF CLINICAL INVESTIGATION
- Toward Gene Therapy for Cystic Fibrosis Using a Lentivirus Pseudotyped With Sendai Virus Envelopes
- (2010) Katsuyuki Mitomo et al. MOLECULAR THERAPY
- A TALE nuclease architecture for efficient genome editing
- (2010) Jeffrey C Miller et al. NATURE BIOTECHNOLOGY
- Single-dose lentiviral gene transfer for lifetime airway gene expression
- (2009) Alice G. Stocker et al. JOURNAL OF GENE MEDICINE
- Generation of Novel AAV Variants by Directed Evolution for Improved CFTR Delivery to Human Ciliated Airway Epithelium
- (2009) Wuping Li et al. MOLECULAR THERAPY
- Dual Reporter Comparative Indexing of rAAV Pseudotyped Vectors in Chimpanzee Airway
- (2009) Terence R Flotte et al. MOLECULAR THERAPY
- Directed evolution of adeno-associated virus to an infectious respiratory virus
- (2009) K. J. D. A. Excoffon et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- The porcine lung as a potential model for cystic fibrosis
- (2008) Christopher S. Rogers et al. AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY
- Generation of a conditional null allele forCftrin mice
- (2008) Craig A. Hodges et al. GENESIS
- Genetic Modification of Adeno-Associated Viral Vector Type 2 Capsid Enhances Gene Transfer Efficiency in Polarized Human Airway Epithelial Cells
- (2008) April F. White et al. HUMAN GENE THERAPY
- Adeno-associated virus–targeted disruption of the CFTR gene in cloned ferrets
- (2008) Xingshen Sun et al. JOURNAL OF CLINICAL INVESTIGATION
- Rapid “Open-Source” Engineering of Customized Zinc-Finger Nucleases for Highly Efficient Gene Modification
- (2008) Morgan L. Maeder et al. MOLECULAR CELL
- Disruption of the CFTR Gene Produces a Model of Cystic Fibrosis in Newborn Pigs
- (2008) C. S. Rogers et al. SCIENCE
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